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NCT05699265Terminated

Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing

Source: ClinicalTrials.gov · Last updated 19 Jun 2025

Reason given: The sponsor withdrew funding since there was no progress with this study for a significant amount of time.

Brief summary (registry, in English)

A longitudinal pilot study will be conducted to determine if there are additional testing modalities that are effective in broadly phenotyping subclinical dysfunction in patients with Fabry disease. Individual patients will undergo serial testing over a two-year period to evaluate for changes in their cardiovasculaorenal function during this period. Novel modalities evaluated will include measures of arterial stiffness, ambulatory blood pressure monitoring, cardiopulmonary exercise testing (CPET), and novel serum and urine biomarkers. The benefit of these measures being evaluated is that they are noninvasive, can be performed rapidly, and have reduced costs compared to the current standard screening modalities. Results from these evaluations will be compared to cMRI and standard urine and serum biomarkers performed clinically per local standard of care. The results will also be compared to both published normative data and data from patients with diabetes mellitus, who have a similar microvascular disease process to patients with Fabry disease.

Status (registry)
Terminated
Study type
Observational
Enrollment
4 (actual)
First submitted
12 Jan 2023
Start date
1 Feb 2023
Primary completion date
1 Apr 2025
Lead sponsor
Children's Hospital Medical Center, Cincinnati
NCT number
NCT05699265

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Other illness

  • Currently prescribed non-ERT treatments for Fabry disease

Other

  • Physical limitation that would preclude exercise

Who the study is looking for

  • Fabry patients with classical disease
  • English speaking, which is needed to assist with obtaining a maximal effort CPET
  • No medical contraindications to cardiopulmonary exercise testing or cMRI
  • Either treatment naïve or current taking ERT

Age in the registry: 8 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

Sites in registry order. Save your city in your profile to sort them by distance.

SiteCityCountrySite status
Cincinnati Children's HospitalCincinnatiUnited StatesUnknown status

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